File 026362
Email Discussion on FDA-Approved Cholesterol Medication Praluent (File 026362)
Email exchange between Jeffrey E. and Sultan Bin Sulayem discussing the FDA approval of Praluent, a new injectable cholesterol-lowering drug, including details about its mechanism, cost, and market implications.
Summary
An email thread initiated on July 24, 2015, discussing the FDA approval of Praluent (developed by Regeneron Pharmaceuticals), a new PCSK9-targeting injectable medication for lowering LDL cholesterol. The discussion references a Forbes article by Matthew Herper detailing the drug's efficacy (58% LDL reduction), high cost ($14,600 annually), target patient populations, and anticipated market impact. The correspondence includes analysis of insurance coverage restrictions, physician preferences, and projected 2020 sales figures.
From: jeffrey E. [jeevacation@gmail.com]Sent: 7/24/2015 8:47:39 PMTo: Sultan Bin SulayemSubject: Re: FDA Approved a Potent and Pricey Cholesterol lowering drug todayyes, it requires a shot, its not a pill and so far no long term effects knownOn Fri, Jul 24, 2015 at 4:37 PM, Sultan Bin Sulayem <Have you heard of this colestrolJul 24, 2015 @ 3:23 PM 1,169 views_ wrote:The FDA Approves A Potent And PriceyCholesterol-Lowering ShotMatthew Herper,Forbes StaffI cover science and medicine, and believe this is biology's century.• Follow on Forbesf1970)Full BioRecent Posts• Popular PostsI believe this is biology's century. I've covered science and medicine for Forbes from the Human GenomeProject through Vioxx to the blossoming DNA technology changing the world today. Email me, follow me onTwitter, circle me onGoogle Plus, or subscribe to my Facebook page.Loading...Loading...The Food and Drug Administration has approved Praluent, a new medicine to lower cholesterol in people whohave established heart disease, for people whose risk of a heart attack or stroke is not being adequatelycontrolled by existing drugs called statins.It might be a big step in the battle against heart disease — and the approval is a triumph for RegeneronPharmaceuticals, the Tarrytown, N.Y.-based biotechnology company that invented it. Praluent is likely to bemore widely used, and to cost more, than Wall Steet analysts expected. That also means it could be a big costHOUSE OVERSIGHT 026362down the line."Obviously, the class is one of the biggest developments in cardiology in the last decade, that will allow us totreat patients who have been extremely difficult to treat with high levels of LDL cholesterol," says StevenNissen, chairman of cardiology at the Cleveland Clinic. "It's very promising."But it will also be very expensive, say Troy Brennan, the chief medical officer of CVS Caremark, the giantpharmacy benefits manager."If these drugs are used as broadly as they could be used, they are going to be byfar the most expensive drug class," Brennan says.Praluent, an injection, lowers low-density lipoprotein, the so-called "bad cholesterol", by as much as 60%, farmore than the statin drugs alone. It is the first drug to work by targeting a protein called PCSK9. Mutations inthe gene that makes it can result in patients having lower cholesterol and a dramatically lower lifetime risk ofheart attacks. A rival medicine from Amgen is also expected to be approved soon. The FDA isclearing Praluent for patients with heterozygous familial hypercholesterolemia, a genetic condition that causeshigh cholesterol, and for people who have heart disease, like those who've had heart attacks or strokes, whoare already taking the maximum dose of a statin they can tolerate.Regeneron and its marketing partner Sanofi say Praluent will have a wholesale cost of $40 a day, or $14,600 ayear, for either of two doses, 75 milligrams or 150 mgs. It's likely that insurance companies will get asignificant discount, because Amgen and Regeneron will compete by giving the insurers rebates.Recommended by ForbesMost PopularPhotos: America's New Manufacturing BoomtownsMost PopularBarrel Rolling With A Thrillionaire EntrepreneurMost PopularPhotos: The 10 Best And Worst States To Make A Living In 2015Trending on LinkedInDonald Trump And The Decline Of American Character--A Cautionary Tale.11174-ta,Most PopularPhotos: America's New Manufacturing BoomtownsMost PopularBarrel Rolling With A Thrillionaire EntrepreneurHOUSE OVERSIGHT 026363Most PopularPhotos: The 10 Best And Worst States To Make A Living In 2015Trending on LinkedInDonald Trump And The Decline Of American Character--A Cautionary Tale"We don't want the noise about these drugs to be price," says Leonard Schleifer, the chief executive andfounder of Regeneron. "We have to make sure that people get access to the drug for a fair price if they'reinsured, for free if they're not insured, or at a discount if they're under-insured."Praluent's price is 46% higher than that forecast by Evercore ISI, an investment bank, and it is being approvedfor a larger group of patients than many Wall Street analysts expected following a meeting convened by theFDA to analyze the data on the drug. Some doctors argued its use should be restricted mainly to patients withFH. But Mark Schoenebaum, Evercore ISI's pharmaceutical analyst, says that investors were initiallydisappointed because they had hoped the FDA would greenlight the drug for an even broader group.Regeneron shares were down 2.5% in afternoon trading before they were halted pending news.But doctors may prescribe the drug more widely than the label suggests. A survey of physicians published thismorning by Geoffrey Porges, an biotechnology analyst at investment bank Sanford C. Bernstein, found thatthey would use the drug in 30% to 40% of patients who had already had heart attacks. He wrote that 2020sales of Praluent could be as high as $4.8 billion.In the survey, doctors expressed a preference for Praluent. One advantage of Regeneron's drug is that doctorswill be able to start it at a lower, 75 milligram dose.Schleifer, who is a medical doctor and, incidentally, a billionaire thanks to his Regeneron stake, says that thecompany expects that as many as 8 million people who don't have their cholesterol well-controlled could becandidates for Praluent. He emphasized, though, that until larger studies prove the drug prevents heart attacksand strokes patients should always choose statins first. It will have sales representatives aInsurance companies and pharmacy benefit managers, who help insurers and employers manage drug costs, aregoing to do their best to restrict use of Praluent, though. Brennan, the CVS Caremark executive, says that heexpects patients will need to have medical records documenting that they have already taken statins. Those nottaking statins would need a blood test confirming that a statin caused elevations in muscle or liver enzymes,not just complaints that they were achy. "We're not trying to replace statins," says Schliefer, the RegeneronCEO.Most people who have muscle problems from statins do not have those elevated muscle enzymes, says Nissen,the Cleveland Clinic cardiologist, who is conducting a clinical trial Amgen and Pfizer, which is alsodeveloping a similar cholesterol drug. He says he has patients who are "desperate" to take a statin but whocannot tolerate it because of muscle pain but do not have elevated muscle enzymes."Dehspite significant progress over the last decades, high cholesterol remains a leading concern in the U.S. andglobally," said Olivier Brandicourt, M.D., Chief Executive Officer, Sanofi. "Praluent demonstrates the powerof the Sanofi and Regeneron alliance to deliver a first-in-class therapy in the U.S. for patients in need. Sanofihas a strong cardiovascular heritage and dedication to these patients, and we look forward to working withother regulatory authorities to make Praluent available to patients worldwide.""We are grateful to the thousands of patients and investigators worldwide who participated in the ODYSSEYclinical trial program," said Leonard S. Schleifer, M.D., Ph.D., Founder, President, and Chief ExecutiveHOUSE OVERSIGHT 026364Officer, Regeneron. "Praluent represents the culmination of more than a decade of tireless work to translate thegenetic-based discovery of PCSK9 into an innovative medicine that brings meaningful value to patients."The approval of Praluent was based on data from the pivotal Phase 3 ODYSSEY program, which showedconsistent, positive results compared to placebo, which included current standard of care therapy (statins). Inthe ODYSSEY LONG TERM trial which evaluated Praluent 150 mg every two weeks, Praluent reduced LDLcholesterol by 58 percent versus placebo at week 24 when added to current standard of care, includingmaximally tolerated statins. In ODYSSEY COMBO I, Praluent 75 mg every two weeks as an adjunct to statinsreduced LDL cholesterol by an additional 46 percent compared to placebo at week 12. At week 24 in the sametrial, Praluent reduced LDL cholesterol by 43 percent compared to placebo. In this study, if additional LDLcholesterol lowering was required based on pre-specified criteria at week 8, Praluent was up-titrated to 150 mgat week 12 for the remainder of the trial. Eighty-three percent of patients remained on their initial 75 mg dose.Praluent is generally well-tolerated with an acceptable safety profile. Local injection site reactions includingredness, itching, swelling or pain/tenderness where the injection is given were the most common events (7.2percent with Praluent versus 5.1 percent with placebo) and resulted in a low discontinuation rate that wascomparable to placebo (0.2 percent with Praluent versus 0.4 percent with placebo). Patients receiving Praluenthad a greater number of injection site reactions, had more reports of associated symptoms, and had reactions oflonger average duration than patients receiving placebo. Other common adverse events occurring morefrequently in patients with Praluent than placebo included symptoms of the common cold and flu or flu-likesymptoms.The companies carefully considered the potential medical value that Praluent offers patients in determining theWholesale Acquisition Cost (WAC). The U.S. WAC price of Praluent is $40 per day ($1,120 every 28 days)for both the 75 mg and 150 mg doses, making Praluent the lowest priced patient-administered monoclonalantibody therapy on an annualized basis. Actual costs to patients, payers and health systems are anticipated tobe lower as WAC pricing does not reflect discounts or rebates. Out-of-pocket costs to patients will varydepending on insurance status and eligibility for patient assistance.Sanofi and Regeneron are committed to ensuring that patients in the U.S. who are prescribed Praluent are ableto access the medicine and receive the support they may need. The companies will launch a comprehensiveprogram that offers support, training and follow up for patients at every step of the process. The program willprovide patient assistance to uninsured or underinsured patients, including providing free medicine to eligiblepatients, and can help identify coverage options for out-of-pocket costs. Additional services includeeducational information, clinical support for physicians, nurses and pharmacists, as well as reimbursementservices, including co-pay support for eligible patients and information about insurance eligibility support. Formore information, please call 1-844-PRALUENT (1-844-772-5836______).Earlier today, the companies announced that the European Medicine Agency's (EMA's) Committee forMedicinal Products for Human Use (CHMP) has adopted a positive opinion for the marketing authorization ofPraluent, recommending its approval for use in certain adult patients with hypercholesterolemia. The EuropeanCommission (EC) is expected to make a final decision on the Marketing Authorization Application forPraluent in the European Union in September 2015.Investor Relations Conference Call on PraluentSanofi and Regeneron will be hosting a conference call for the financial community on Praluent® (alirocumab)Injection for the treatment of high LDL cholesterol in adult patients. The conference call will take place onFriday, July 24, 2015 (21:30 CET / 20:30 BST/ 15:30 EDT / 12:30 PDT).The call will be available through audio webcast at www.sanofi.com and www.regeneron.com and also via thefollowing telephone numbers:France:+33 (0) 1 70 80 71 53_HOUSE OVERSIGHT 026365UK:+44 (0) 203 107 0289U.S.:+1 888 660 6127Access code: 81662098ODYSSEY ProgramThe ODYSSEY Phase 3 program is one of the most comprehensive clinical trial programs ever conducted foran investigational LDL cholesterol lowering therapy. The program includes 14 global Phase 3 trials evaluatingmore than 23,500 patients. The primary efficacy end point in all of the studies was the mean percent reductionfrom baseline in LDL cholesterol at week 24 compared to placebo (maximally tolerated statin therapy); all ofthe completed studies met their primary endpoint. A significantly higher proportion of patients achieved LDLcholesterol of less than 70 mg/dL in the Praluent group as compared to placebo at both week 12 and week 24.The ongoing ODYSSEY OUTCOMES trial will prospectively evaluate the cardiovascular benefits of Praluentin approximately 18,000 patients.Important Safety InformationDo not use PRALUENT if you are allergic to alirocumab or to any of the ingredients in PRALUENT.Before you start using PRALUENT, tell your healthcare provider about all your medical conditions, includingallergies, and if you are pregnant or plan to become pregnant or if you are breastfeeding or plan to breastfeed.Tell your healthcare provider or pharmacist about any prescription and over-the-counter medicines you aretaking or plan to take, including natural or herbal remedies.PRALUENT can cause serious side effects, including allergic reactions that can be severe and requiretreatment in a hospital. Call your healthcare provider or go to the nearest hospital emergency room right awayif you have any symptoms of an allergic reaction including a severe rash, redness, severe itching, a swollenface or trouble breathing.The most common side effects of PRALUENT include: redness, itching, swelling, or pain/tenderness at theinjection site, symptoms of the common cold, and flu or flu-like symptoms. Tell your healthcare provider ifyou have any side effect that bothers you or that does not go away.Talk to your doctor about the right way to prepare and give yourself a PRALUENT injection and follow the"Instructions for Use" that comes with Praluent.You are encouraged to report negative side effects of prescription drugs to the FDA. Visitwww. fda. gov/medwatch or call 1 -800-FDA-1088______Please click here for the full Prescribing Information.About SanofiSanofi, an integrated global healthcare leader, discovers, develops and distributes therapeutic solutions focusedon patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetessolutions, human vaccines, innovative drugs, consumer healthcare, emerging markets, animal health and thenew Genzyme. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).please noteThe information contained in this communication isconfidential, may be attorney-client privileged, mayconstitute inside information, and is intended only forthe use of the addressee. It is the property ofHOUSE OVERSIGHT 026366JEEUnauthorized use, disclosure or copying of thiscommunication or any part thereof is strictly prohibitedand may be unlawful. If you have received thiscommunication in error, please notify us immediately byreturn e-mail or by e-mail to jeevacation@gmail.com, anddestroy this communication and all copies thereof,including all attachments. copyright -all rights reservedHOUSE OVERSIGHT 026367